AAVantgarde Bio has closed a $141 million (€122 million) Series B financing to advance its lead clinical programs targeting inherited retinal diseases. The financing round was co-led by Schroders Capital, Atlas Venture, and Forbion, with participation from new investors including Amgen Ventures, Athos KG, CDP Venture Capital, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors, as well as continued support from Longwood Fund and Sofinnova Partners.
The clinical stage biotechnology company is focused on developing gene therapies for Stargardt disease and retinitis pigmentosa secondary to Usher syndrome type 1B, two conditions for which there are currently no approved treatments.
The proceeds will support progression of the AAVB-039 CELESTE proof-of-concept study in Stargardt disease, a condition caused by mutations in the ABCA4 gene that leads to progressive loss of central vision. The funding will also support the completion of the STELLA natural history study, which includes more than 100 patients. In addition, the Series B enables continued advancement of AAVB-081 LUCE, a phase 1/2 trial targeting retinitis pigmentosa secondary to Usher 1B caused by mutations in MYO7A. Usher syndrome 1B results in combined hearing and vision loss, creating a substantial impact on the patient’s quality of life.
AAVantgarde’s approach utilizes gene augmentation to deliver full-length, functional versions of ABCA4 and MYO7A, targeting the underlying genetic causes rather than managing symptoms. By restoring these proteins, the programs aim to help preserve or improve retinal function across a broad range of patients regardless of specific mutation subtype.
The financing marks a significant step forward in advancing therapies for conditions that currently lack viable treatment options and are a leading cause of visual impairment in children and young adults. The company continues to strengthen its clinical development capabilities as these programs move closer to generating meaningful clinical data for patient communities affected by these rare and progressive diseases.
KEY QUOTES:
“This investment is a strong endorsement of our team, our science, and two clinical IRD programs. Both programs address the root genetic causes of devastating conditions and offer hope of improvement to patients and families living with progressive vision loss.”
Dr. Natalia Misciattelli, CEO of AAVantgarde
“We are proud to support AAVantgarde at this important stage of clinical development. Its innovative platform could enable best-in-class treatments for inherited eye diseases like Usher 1B and Stargardt’s by overcoming key barriers in current AAV technologies and reaching patients who currently have limited treatment options.”
Harry Raikes, Head of UK Venture Investments at Schroders Capital
“Inherited Retinal Disorders (IRDs), such as Stargardt disease and RP secondary to Usher 1B, are among the leading causes of blindness in children and young adults worldwide. This financing milestone represents a critical step forward in our ability to bring hope to patients affected by Usher 1B and Stargardt disease, two hereditary retinal disorders with urgent, unmet need and provides the potential to help these underserved patient populations and improving their lives.“
Dr. Peter Kaiser, Professor of Ophthalmology at the Cleveland Clinic Lerner College of Medicine and AAVantgarde Board Member
